IV methylprednisolone pulses for infants with NEHI

Efficacy of Methylprednisolone Pulses in Neuroendocrine Celles Hyperplasia of Infancy : An Early Phase Study

This study will try short courses of IV methylprednisolone to see if they shorten how long infants under 12 months with NEHI need oxygen.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: N/A to 12 Years
• Sex: All
• Sponsor: Assistance Publique - Hôpitaux de Paris (Academic / other)
• Locations: 1 site (Paris)
• Trial ID: NCT06471556 on ClinicalTrials.gov

What this trial studies

This is a Phase 2, single‑arm A'Hern single‑stage study that will enroll 18 hypoxic infants under 12 months with a clinical and CT‑based diagnosis of NEHI who are followed in RespiRare centers. All participants receive up to six IV methylprednisolone pulses given as three‑day daily infusions (approximately 10 mg/kg/day) spaced about four weeks apart, with cardiorespiratory monitoring during infusions. The main goal is to see whether these pulses allow stopping or reducing supplemental oxygen more quickly than expected under usual care. Twelve clinic/hospital visits are scheduled for screening, pulse administrations, and follow‑up over the treatment period to record oxygen needs and safety data.

Who should consider this trial

Good fit: Infants under 12 months with NEHI confirmed by CT pattern (and/or biopsy) who require oxygen and are followed at a participating RespiRare center are the intended participants.

Not a fit: Children with other causes of childhood interstitial lung disease, prior IV methylprednisolone pulses, diabetes, or those older than 12 months are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, the treatment could shorten infants' dependence on supplemental oxygen and reduce hospitalizations and feeding support needs.

How similar studies have performed: High‑quality evidence for pulse IV corticosteroids specifically in NEHI is lacking; steroids have been used empirically in some chILD cases but this pulse approach is largely untested in NEHI.

Eligibility criteria

Inclusion Criteria:

* Infant aged under 12 months
* NEHI diagnosis based on:

  * The recently validated clinical Liptzin score ≥7/10 associated with a suggestive thoracic CT pattern with ground glass opacities confined to middle lobe, lingula, and paramediastinal lung areas OR
  * a clinical and thoracic CT suspicion and a lung biopsy showing an increased number of neuroendocrine cells in the epithelial airways area (at least one bronchiole with at least 10% of neuroendocrine cells)
* Oxygen requirement (awake and/or asleep) based on the usual pediatric recommendations (see section 4.1.1)
* Followed in one of the RespiRare participating centers
* Written informed consent of the holder(s) of its legal representative at the inclusion

Exclusion Criteria:

* Other cause of chILD assessed by lab biology tests, genetic analysis for surfactant genes (if available), bronchoalveolar lavage, and/or lung biopsy.
* Patient treated with IV methylprednisolone pulses before (any time)
* Diabetes
* Uncontrolled arterial hypertension
* Absence of Health care insurance
* Ongoing infection
* Immunization with a live attenuated vaccine within the past two weeks
* Long term treatment with Azithromycin and/or Hydroxychloroquine
* Patients already included in an interventional study (RIPH1, clinical investigation or clinical trial)

Where this trial is running

Paris

• Pediatric Pulmonology Department and Reference centre for rare lung diseases — Paris, France (Recruiting)

Study contacts

• Principal investigator: Nadia NATHAN — Assistance Publique - Hôpitaux de Paris
• Study coordinator: Nadia NATHAN

Conditions Neuroendocrine Cell Hyperplasia of Infancy; neuroendocrine cells hyperplasia of infancy; Persistent tachypnoea of infancy; Childhood interstitial lung diseases; IV Corticosteroids pulses; Methylprednisolone; Oxygen therapy